To generate the variety of cell types found throughout our bodies, progenitor cells undergo a specification process termed differentiation. Appropriate cellular differentiation is essential for both embryonic development and adult tissue homeostasis, whereas misregulation of these pathways is commonly observed in many human diseases. Recent work suggests that progenitor cells and their differentiated counterparts express variations of the same protein through a process called alternative splicing. By characterizing the regulation of alternative splicing during differentiation, the proposed research will enhance our understanding of the regulatory networks governing normal cellular biology and may uncover novel therapeutic opportunities for a variety of human diseases.

Statement of Benefit to California:

This project has the potential to benefit both the economy of the state of California as well as the health care provided to residents. In the successful study of stem cells and how they differentiate, we hope the results will lead to new therapeutic strategies, diagnostics, and cures for diseases (specifically in the area of neurodegenerative diseases). These new therapies would significantly impact the care given to California residents with these diseases. Clinicians will better understand the disease of their patients and be able to tailor care for them. As there is much room for growth in the area genetic therapeutics, we believe new discoveries in this area would be a catalyst for the creation of biotechs in [REDACTED]. Also, we are requesting funds from CIRM which will provide salaries for several full-time employees to a number of people who reside in California as well as purchasing the majority of our supplies from area companies.